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Completed

NCT Number: NCT03390985

Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor

Specific aims:

1. To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and nutritional status 2. To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity, 3. To assess altered CFTR function using new available in vivo tests, 4. To validate newly developed in vivo sweat tests with well established functional tests, 5. To establish correlation between the CFTR response to Vx-770 measured in a new ex vivo method (organoids) and the actual clinical and/or functional response in individual patients, 6. To examine response in other CF-specific features such as aqua wrinkling. 7. To examine if sleep/activity level changes. 8. To establish a biorepository to enable further investigations.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • any patient being prescribed ivacaftor

Exclusion criteria

  • only relates to specific tests

Treatment and study plan

Primary outcomes

  1. Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements

    Time frame: 7 study visits over a 2 year period

    NPD is measured to assess CFTR function as difference in mV between post and pre-drug start

  2. Functional in vivo test: Beta-adrenergic sweat secretion test

    Time frame: 7 study visits over a 2 year period

    The beta-adrenergic sweat secretion test is measured as ratio of beta-adrenergic/cholinergic sweat secretion as difference between post and pre-drug start

  3. Functional in vitro test: Rectal tissue biopsies to measure CFTR function in vitro using Ussing chamber studies and organoids

    Time frame: one study visit

    Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro

  4. Functional in vitro test: Collect nasal brushes for CFTR to generate nasal cell cultures for the assessment of ivacaftor response and CFTR function

    Time frame: 2 study visits in 6 months

    Nasal cultures are used to measure the response to ivcaftor in vitro as difference of the forskolin-induced response of CFTR to ivacaftor

Secondary outcomes

  1. Measurement of aqua wrinkling

    Time frame: 2 study visits in 3 months

    CF patients response to submerge in water for 5 min to develop a skin phenomenon known as aqua wrinkling is being measured subjectively pre and post-drug

  2. Measurement of exhaled fractionated nitrogen oxide (FeNo)

    Time frame: 5 study visits in 1 year

    the fraction of the exhaled NO in % In the expiration of CF patients is being measured pre-and post-drug

  3. Measurements of the lung clearance index

    Time frame: 5 study visits in 1 year

    Multiple breath wash-out technique is being used to measure the lung clearance index (no unit) pre and post-drug

  4. Measurements of the pancreas function using fecal elastase test

    Time frame: 4 study visits in 2 years

    Fecal elastase in stool is determined in microg/g stool pre and post-drug

  5. Measurements of the pancreas function using serum trypsinogen

    Time frame: 4 study visits in 2 years

    Serum trypsinogen is determined in nmol/L pre and post-drug

  6. Assessment of the nutritional status

    Time frame: 4 study visits in 2 years

    Nutritional status is assessed using the BODPOD in kg fat or non-fat mass change or percentage fat or non-fat mass change pre and post-drug

  7. Measurements of the resting energy expenditure

    Time frame: 4 study visits in 2 years

    The resting energy expenditure is measured using indirect calorimetry in kcal/d pre and post-post

  8. Assessment of pulmonary radiological changes using high resolution CT

    Time frame: 4 study visits in 2 years

    Radiological pulmonary changes are being assessed using high resolution CT pre and post-drug (descriptive and Bhalla score)

  9. Assessment of glucose tolerance

    Time frame: 4 study visits in 2 years

    Glucose tolerance is being assessed using the organ glucose tolerance test and glucose levels 2 hrs post in mmol/L will be compared pre and post-drug

Sponsors and collaborators

Lead sponsor

Tanja Gonska

Other

Registry information

Acronym: G551D

Important dates

Study start
2013
Primary completion
2017
Study completion
2017
First posted
Jan 5, 2018
Registry last updated
Jan 5, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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